Muscle dystrophies (MDs) are a heterogeneous group of inherited human disorders primarily affecting skeletal muscles. Typically, MDs show diffuse wasting and weakness of muscles, associated with degeneration and regeneration of muscle fibers. Duchenne MD is the most common and severe form of MD worldwide. This progressive and lethal X-linked myopathy is characterized by a deficiency of dystrophin, a subsarcolemmal protein critical in membrane stabilization and prevention of contraction-induced cell membrane damage (Durbeej, M. and Campbell, K. P. (2002) Curr. Opin. Genet. Dev., 12, 349-361).
There is no known cure for muscular dystrophy (MD). Inactivity (such as bed rest and even sitting for long periods) can worsen the disease. Physical therapy, occupational therapy, orthotic intervention (e.g., ankle-foot orthosis), speech therapy and orthopedic instruments (e.g., wheelchairs and standing frames) may be helpful.
There is no specific treatment for any of the forms of MD. Physical therapy to prevent contractures and maintain muscle tone, orthoses (orthopedic appliances used for support) and corrective orthopedic surgery may be needed to improve the quality of life in some cases. Thus, there is a need in the art for improved methods of treatment for MDs.